Japanese MHLW Grants Orphan Drug Designation in Japan to Takeda's Oral Proteasome Inhibitor Ixazomib for Patients with Relapsed/Refractory Multiple Myeloma

Osaka, Japan, February 26, 2016 --- Takeda Pharmaceutical Company Limited (TSE: 4502) today announced that the Japanese Ministry of Health, Labour and Welfare has granted Orphan Drug designation* to ixazomib (generic name, development code: MLN9708), an investigational oral proteasome inhibitor for the treatment of patients with relapsed and/or refractory multiple myeloma. The Orphan Drug designation is a system for supporting and promoting the development of drugs that are not sufficiently researched and developed due to a small number of patients, regardless of high medical need.

Ixazomib's phase 3 trials for patients with multiple myeloma are now being conducted in Japan. The drug became available in the U.S., under the brand name NINLARO^®, in December, 2015 after the U.S. Food and Drug Administration (FDA) approval in combination with lenalidomide and dexamethasone as a treatment for patients with multiple myeloma who have received at least one prior therapy. The drug is currently under review in the European Union. The European Medicines Agency (EMA) granted ixazomib an accelerated assessment in July 2015 and accepted its Marketing Authorization Application (MAA) in August 2015 for the treatment of patients with relapsed and/or refractory multiple myeloma.

This Orphan Drug designation reflects the high medical need for oral proteasome inhibitor ixazomib as a potential new treatment option for patients with multiple myeloma in Japan, and further demonstrates Takeda’s commitment to putting the patient at the center. Takeda will continue its development activities so that this innovative drug can be delivered to Japanese patients as soon as possible.

* The designation is granted by the Minister of Health, Labour and Welfare for drugs that meet the designation criteria, which include the following: the number of patients who may use the drug is less than 50,000 in Japan; there is no alternative appropriate drug or treatment; high efficacy or safety is expected compared to existing products; there is a theoretical rationale for using the product for the target disease and the development plan is appropriate. If a product is designated as an orphan drug, support such as subsidies, guidance and consultation on research and development activities by the Minister of Health, Labour and Welfare, preferential tax treatment, priority review, and extension of re-examination period will become available.

About ixazomib and the TOURMALINE Trials
Ixazomib (MLN9708) is an investigational oral proteasome inhibitor which is being studied in multiple myeloma, systemic light-chain (AL) amyloidosis, and other malignancies. It is the first oral proteasome inhibitor to enter Phase 3 clinical trials.

Ixazomib was granted orphan drug designation in multiple myeloma in both the U.S. and Europe in 2011 and for AL amyloidosis in both the U.S. and Europe in 2012. Ixazomib received Breakthrough Therapy status by the U.S. FDA for relapsed or refractory systemic light-chain (AL) amyloidosis, a related ultra orphan disease, in 2014. Ixazomib was launched in the U.S. in December, 2015 under the brand name NINLARO^®, and is also currently under review by the European Medicines Agency for the treatment of patients with relapsed and/or refractory multiple myeloma.

The comprehensive ixazomib clinical development program, TOURMALINE, further reinforces Takeda’s ongoing commitment to developing innovative therapies for people living with multiple myeloma worldwide and the healthcare professionals who treat them. TOURMALINE includes a total of five ongoing pivotal trials – four investigating every major multiple myeloma patient population and one in light-chain amyloidosis:

• ž  TOURMALINE-MM1, investigating ixazomib vs. placebo, in combination with lenalidomide and dexamethasone in relapsed and/or refractory multiple myeloma. This trial is currently ongoing, and patients continue to be treated to progression and will be evaluated for long-term outcomes.
• ž  TOURMALINE-MM2, investigating ixazomib vs. placebo, in combination with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma
• ž  TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma following induction therapy and autologous stem cell transplant (ASCT)
• ž  TOURMALINE-MM4, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma who have not undergone ASCT
• ž  TOURMALINE-AL1, investigating ixazomib plus dexamethasone vs. physician choice of selected regimens in patients with relapsed or refractory AL amyloidosis

About Multiple Myeloma
Multiple myeloma is a cancer of the plasma cells, which are found in the bone marrow. In multiple myeloma, a group of monoclonal plasma cells, or myeloma cells, becomes cancerous and multiplies. These malignant plasma cells have the potential to affect many bones in the body, possibly resulting in compression fractures, lytic bone lesions and related pain. Multiple myeloma can cause a number of serious health problems affecting the bones, immune system, kidneys and red blood cell count, with some of the more common symptoms including bone pain and fatigue, a symptom of anemia. Multiple myeloma is a rare form of cancer, with 114,000 new cases globally per year. It is reported that there are approximately 14,000 patients with multiple myeloma in Japan.

Media Contacts:
Tsuyoshi Tada
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